Indlela Ucwaningo Nokuthuthukiswa Kwezidakamizwa Zezintandane Kunconywa
Iyini isidakamizwa sezintandane nokuthi yimuphi umthetho we-Orphan Drug Act? Kungani ucwaningo nentuthuko yalezi zidakamizwa zibalulekile futhi yiziphi izithiyo?
Iyini i-Orphan Drug? - Incazelo
Isidakamizwa sezintandane imithi (imithi) ehlala ingathuthuki ngenxa yokuntuleka kwenkampani ukuthola inzuzo yemithi. Ngokuvamile isizathu sokuthi lesi sidakamizwa asizuzi ukuthi kunabantu abambalwa abazokuthenga lesi sidakamizwa uma kulinganiswa nokucwaninga nokuthuthukiswa okudingekayo ukukhiqiza lesi sidakamizwa.
Ngamazwi alula, izidakamizwa zezintandane yizo izinkampani ezingalindele ukwenza imali eningi, futhi esikhundleni salokho ziqondisa imizamo yazo ezidakamizwa ezizoletha imali.
Okwenza Abanye Izidakamizwa "Ziyizintandane"
Izinkampani zokwelapha (izidakamizwa) nezinkampani ze-biotech ziqhubeka zicwaninga futhi zithuthukisa imishanguzo emisha yokwelapha izimo zezokwelapha, futhi izidakamizwa ezintsha zifika emakethe njalo. Abantu ababhekene nezifo ezingavamile noma ukuphazamiseka, ngakolunye uhlangothi, aboni ukuhlola okufanayo kwezidakamizwa ngezifo zabo. Lokhu kungenxa yokuthi izinombolo zabo zincane futhi ngakho-ke imakethe engaba khona yezidakamizwa ezintsha zokwelapha lezi zifo ezingavamile (ngokuvamile ezibhekwa ngokuthi "izidakamizwa zezintandane") nazo zincane.
Isifo esingavamile senziwa ngabantu abangaphansi kuka-200,000 e-United States noma abangaphansi kuka-5 ngabangu-10 000 abantu base-European Union. Izinhlaka zikahulumeni zokulawula e-United States naseYurophu Union ziye zathatha izinyathelo zokunciphisa lokhu okungafani nokuthuthukiswa kwezidakamizwa
Izikhuthazo zokwandisa ukuthuthukiswa kwezidakamizwa zezintandane
Ukuqaphela ukuthi izidakamizwa ezanele ngezifo ezingavamile ezingakaze zakhiwe e-US kanti lezo zinkampani ezidakamizwa ziyoholela ekulahlekelweni kwezimali ekuthuthukiseni izidakamizwa ezimweni ezingavamile, i-US Congress yadlulisa uMthetho we-Orphan Drug Act ngo-1983.
Ihhovisi lase-US Lokuthuthukiswa Komkhiqizo Wezintandane
I-US Food and Drug Administration (FDA) inesibopho sokuqinisekisa ukuphepha nokusebenza kwemithi emakethe e-United States.
I-FDA yasungula iHhovisi leNtuthuko yomkhiqizo wezintandane (OOPD) ukusiza ekuthuthukiseni izidakamizwa zezintandane (neminye imikhiqizo yezokwelapha ngezifo ezingavamile), kufaka phakathi ukunikeza izibonelelo zocwaningo.
Izidakamizwa zezintandane, njengezinye izidakamizwa, zitholakala ziphephile futhi ziphumelele ngokucwaninga nokuhlolwa kwemitholampilo ngaphambi kokuba i-FDA izobavumela ukuthengisa.
Umthetho We-Orphan Drug Act ka-1983
Umthetho we-Orphan Drug Act unikeza amandla okukhuthaza izinkampani ukuba zihlakulele izidakamizwa (nezinye imikhiqizo yezokwelapha) ezimakethe ezincane zabantu abanezinkinga ezingavamile (e-US, amaphesenti angu-47 ezinkinga ezingavamile ezingathinta abantu abangaphansi kuka-25 000). Lezi zikhuthazo zihlanganisa:
- Izikweletu zentela zikahulumeni zocwaningo olwenziwe (kuze kube yizindleko ezingamaphesenti angu-50) ukuthuthukisa izidakamizwa zezintandane.
- Ukuzimela komuntu oneminyaka engu-7 ekuthengiseni izidakamizwa kwenkampani yokuqala ukuthola imvume yokumaketha ye-FDA yomuthi othile. Lokhu kusebenza kuphela ekusetshenzisweni okuvunyelwe kwesidakamizwa. Olunye uhlelo lokusebenza oluhlukile lungabuye lwavunyelwa yi-FDA, futhi inkampani izoba namalungelo okuthengisa okukhethekile okudakamizwa ukuze kusetshenziswe.
- Izimali zokukhokhelwa kwezicelo zokuvunywa kwezidakamizwa kanye nemali ye-FDA yomkhiqizo wonyaka.
Ngaphambi kokuya komthetho we-Orphan Drug Act, izidakamizwa ezimbalwa zezintandane zitholakalayo ukuphatha izifo ezingavamile.
Kusukela eMthethweni, izidakamizwa ezingaphezu kuka-200 zezintandane ziye zavunywa yi-FDA yokuthengisa e-US
Umthelela we-Orphan Drug Act ku-US Pharmaceuticals
Njengoba uMthetho Wezidakamizwa Wama-Orphan waba ngo-1983, kuye kwaba nomthelela ekuthuthukiseni izidakamizwa eziningi. Ngo-2012 bekukhona okungenani izidakamizwa ezingama-378 ezivunyiwe ngale nqubo, futhi inombolo iyaqhubeka ukukhuphuka.
Isibonelo sezidakamizwa ezitholakala ngenxa yomthetho wezidakamizwa zezintandane
Phakathi kwezidakamizwa ezivumile zihlanganisa lezo ezifana nalezi:
- I-hormone ye-Adrenocorticotropic (ACTH) yokwelapha ama- spasms amancane
- I-Tetrabenzine yokwelapha i-chorea eyenzeka kubantu abanesifo sikaHuntington
- Ukwelapha esikhundleni se-enzyme kulabo abanenkinga yokugcina iglycogen, isifo sikaPompe
Ucwaningo Lwezwe Lonke Nokuthuthukiswa Kwezidakamizwa Zezintandane
Njenge-US Congress, uhulumeni wase-European Union (EU) uye waqaphela isidingo sokwandisa ucwaningo nokuthuthukiswa kwezidakamizwa zezintandane.
IKomidi leMithi yokwelapha yezintandane
Isungulwe ngo-1995, i-European Medicines Agency (EMEA) inesibopho sokuqinisekisa ukuphepha nokusebenza kwemithi emakethe e-EU. Ihlanganisa ndawonye izinsizakalo zesayense zamazwe angama-25 e-EU. Ngo-2000, iKomidi leMithi yokwelapha ye-Orphan (COMP) yasungulwa ukuze libheke ukuthuthukiswa kwezidakamizwa zezintandane e-EU.
Umthethonqubo weMithi Yezintandane
Umthethonqubo weMithi Yezintandane, eyadluliselwa yiMkhandlu waseYurophu, uhlinzeka ngokugqugquzela izidakamizwa zezintandane (neminye imikhiqizo yezokwelapha ngezifo ezingavamile) e-EU, kufaka phakathi:
- Ukukhishwa kwezimali eziphathelene nenqubo yokugunyazwa kokumaketha.
- Ukuzimela komuntu oneminyaka engu-10 ekuthengiseni izidakamizwa kwenkampani yokuqala ukuthola ukugunyazwa kwemakethe ye-EMEA. Lokhu kusebenza kuphela ekusetshenzisweni okuvunyelwe kwesidakamizwa.
- Ukugunyazwa kokukhangisa komphakathi - inqubo eyinhloko yokugunyazwa kokuthengisa okudluliselwa kuwo wonke amazwe e-EU.
- Usizo lwe-Protocol, okusho ukunikezwa iseluleko sezesayensi ezinkampanini zezidakamizwa mayelana nokuhlolwa okuhlukahlukene kanye nokuvivinywa komtholampilo okudingekayo ukuba kwenziwe izidakamizwa.
Umthethonqubo weMithi yokwelapha yezintandane uye waba nethonya elifanayo elizuzayo e-EU ukuthi uMthetho we-Orphan Drug Act wayese-US, ukwandisa kakhulu ukuthuthukiswa nokukhangiswa kwezidakamizwa zezintandane ngenxa yezifo ezingavamile.
Ngaphansi kwe-Orphan Drug Act
Ngaleso sikhatsi kunengxabano enkulu phezu kwe-Orphan Drug Act, ngesidingo sokwelashwa kwezifo ezingavamile ngakwesinye kwezinga, kanye nemibono mayelana nokusimama komunye. Ngokujabulisayo, lezi zenzo, kokubili e-United States naseYurophu ziye zaphakamisa ukuqwashisa izifo eziningi ezingavamile, okuthi, uma zengezwe ndawonye, akuzona zonke izinto ezingavamile.
Imithombo:
Herder, M. Iyini Inhloso Ye-Orphan Drug Act? . PLoS Medicine . 2017. 14 (1): e1002191.
Murphy, S., Puwanant, A., no R. Griggs. Imiphumela engavumelekile yokuqokwa komkhiqizo wezintandane ngenxa yezifo eziningana ze-Neurological Disorders. Ama-Annal of Neurology . 2012. 72 (4): 481-490.
Ukuphathwa kwe-US nokuDrug. Ukuklama Umkhiqizo Wezintandane: Izidakamizwa Nemikhiqizo Yezinto Eziphilayo. Kubuyekezwe 05/02/16. https://www.fda.gov/forindustry/developingproductsforrarediseasesconditions/howtoapplyfororphanproductdesignation/default.htm